MC Exclusive | Hospitals to set up panels to vet requests for aid to treat rare diseases

The Centre has asked designated hospitals that treat rare diseases to set up expert panels that will decide on a grant of Rs 50 lakh within a month of receiving an application for financial aid from a patient.

The directive is part of the guidelines for granting financial assistance to patients suffering from rare diseases. The Union health ministry issued the guidelines last week under the National Policy for Rare Diseases, 2021.

The government has increased the grant amount and widened the eligibility of patients who can apply for financial aid. It said every request for a grant processed by the expert panels at specified centres of excellence will be considered by the ministry before financial aid is issued.

Two months ago, the Centre increased the limit of financial support to Rs 50 lakh from Rs 20 lakh for a certain category of rare-disease patients eligible under the Pradhan Mantri Jan Aarogya Yojana, the government’s flagship health insurance scheme.

But now the norms have been liberalised and all rare-disease patients in India are eligible for a one-time grant of as much as Rs 50 lakh from the Centre under the National Policy for Rare Diseases (NPRD), provided the special committees at the centres of excellence approve their applications.

With the detailed guidelines issued by the ministry now, a large number of patients can benefit in the coming months, said Aakash Shukla, the nodal officer for rare diseases at King Edward Medical College in Mumbai.

The clarity provided by the government to the centres of excellence is a welcome and much-needed development, said Saurabh Singh, cofounder of the Rare Disease India Foundation.

The health ministry recognises eight such centres of excellence including All India Institute of Medical Sciences and Maulana Azad Medical College in New Delhi, Sanjay Gandhi Post Graduate Institute of Medical Sciences in Lucknow, King Edward Medical College in Mumbai, and Centre for DNA Fingerprinting and Diagnostics with Nizam’s Institute of Medical Sciences in Hyderabad.

The other recognised centres are Post Graduate Institute of Medical Education and Research in Chandigarh, Institute of Post Graduate Medical Education and Research in Kolkata, and Centre for Human Genetics with Indira Gandhi Hospital in Bengaluru.

Rare diseases are mostly life-threatening and chronic. The World Health Organization defines a rare disease as a debilitating, lifelong condition with a prevalence of 1 or less per 1,000 population.

Drugs or cures for many rare diseases are not easily available, and when they are, they are prohibitively expensive, making treatment or procedures unaffordable for the majority of patients in India.

The government came up with the NPRD last year, proposing a grant of up to Rs 20 lakh for patients with diseases listed in Group 1, which are disorders amenable to a one-time curative treatment with stem cell transplantation or organ transplantation.

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Category 2 rare diseases are those that require long-term or lifelong treatment with relatively low cost. These can be managed with special dietary formulae or food for special medical purposes and include conditions amenable to hormones or specific drugs.

Group 3 rare diseases include those for which definitive treatment may be very high-cost and needed lifelong.

The provision of Rs 20 lakh aid to only Group 1 patients was revised in June this year.

As part of the policy, the specified hospitals, in collaboration with the Indian Council of Medical Research, must prepare a registry for rare diseases in India. Some estimates suggest there are as many as 70 million to 80 million such patients in the country.

The latest guidelines say that while the ministry will provide support of up to Rs 50 lakh to patients in need, Group 2 patients will be eligible to get support from their state governments.

“Keeping in view the resource constraint and compelling health priorities, it will be difficult for the government to fully finance the treatment of high-cost rare diseases,” it said. “The gap will be filled up by providing financial assistance through crowdfunding.”

So far, almost 350 patents have registered on the crowdfunding portal that the government started last year. But in the absence of clear-cut guidelines, the centres of excellence are yet to fully utilise the facility.

The guidelines also encourage hospitals for rare diseases to seek funds for patients through corporate social responsibility allocations of companies, drug manufacturers and other agencies.

The ministry said additional centres of excellence can be set up depending on regional requirements. If a disease has been excluded from the classifications, these centres can appeal for their addition with supporting scientific evidence.

“We get a lot of patients from all across the country who are in immediate need of treatment and this number has gone up since NPRD 2021 came into being,” said Dr Akash Shukla of King Edward Medical College.

Dr Shubha Phadke, who heads the rare disease cell at SGIMER in Lucknow, said some clarifications are needed before the institute can start processing requests for financial aid.

“We need to understand the nitty-gritty of how the funds will be received from the health ministry and utilised for treatment,” she said.

“Considering the delay so far, we would now urge the centres of excellence to expedite the process of starting treatment for all eligible patients, particularly those diagnosed with ultra-rare genetic conditions, for which approved therapies have been available in India and for which sufficient evidence of good clinical outcomes exists,” said Saurabh Singh, chairman and co-founder of the Rare Disease India Foundation.